Success of gene therapy for a form of inherited blindness depends on timing

Nearly two decades ago, a gene therapy restored vision to Lancelot, a Briard dog who was born with a blinding disease. This ushered in a period of hope and progress for the field of gene therapy aimed at curing blindness, which culminated in the 2017 approval of a gene therapy that improved vision in people with Leber congenital amaurosis (LCA), a rare, inherited form of blindness closely related to the condition seen in Lancelot. It represents the first FDA-approved gene therapy for an inherited genetic disease.